Mai ElMallah
Associate Professor of Pediatrics
Our laboratory focuses on the control of breathing and pulmonary mechanics in murine models of several genetic diseases. We are currently focuased on ALS mouse models and the Pompe disease mouse model. We examine the impact of gene therapy and neuromodulation on respiratory insufficiency that results from neuromuscular weakness. As a clinician scientist, my goal is to bring therapy from the bench to the bedside and enhance our research at the bench through observations at the bedside.
Current Appointments & Affiliations
- Associate Professor of Pediatrics, Pediatrics, Pulmonary and Sleep Medicine, Pediatrics 2018
Contact Information
- Background
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Education, Training, & Certifications
- Pediatric Pulmonary Fellowship, Pediatrics, University of Florida College of Medicine 2010 - 2012
- Residency, Pediatrics, Baylor College of Medicine 2002 - 2005
- Junior House Officer, Queen Elizabeth Hospital, England 2001 - 2002
- M.B.B.Ch., Queens University Belfast (N. Ireland) 2001
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Duke Appointment History
- Instructor in the Department of Pediatrics, Pediatrics, Pulmonary and Sleep Medicine, Pediatrics 2017 - 2018
- Expertise
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Subject Headings
- Research
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Selected Grants
- Targeted Therapy for Pompe Disease awarded by National Institutes of Health 2019 - 2024
- Epigenome Editing Technologies for Treating Diverse Disease awarded by National Institutes of Health 2019 - 2023
- Award for a Pharmacist 2019 - 2022
- Mental Health Screening in Duke Pediatric CF Patients 2018 - 2021
- Evolving High Potency AAV Vectors for Neuromuscular Genome Editing awarded by National Institutes of Health 2018 - 2021
- Evolving High Potency AAV Vectors for Neuromuscular Genome Editing awarded by National Institutes of Health 2018 - 2021
- A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease awarded by National Institutes of Health 2017 - 2021
- VX16-809-120 2018 - 2021
- Respiratory Characterization of DMD Mouse Models 2019 - 2020
- Cystic Fibrosis Foundation 2019 - 2020
- Sav005-04 2017 - 2019
- Cystic Fibrosis Foundation 2018 - 2019
- Gene Therapy for Upper Airway and Respiratory Insufficiency in an ALS Mouse Model awarded by National Institutes of Health 2016 - 2019
- Parker B. Francis Jo Rae Wright Award for Scientific Excellence 2018 - 2019
- Novel Approaches for Correcting Respiratory Insufficiency in Pompe Disease awarded by National Institutes of Health 2014 - 2019
- Publications & Artistic Works
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Selected Publications
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Academic Articles
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McCall, Angela L., and Mai K. ElMallah. “Macroglossia, Motor Neuron Pathology, and Airway Malacia Contribute to Respiratory Insufficiency in Pompe Disease: A Commentary on Molecular Pathways and Respiratory Involvement in Lysosomal Storage Diseases..” Int J Mol Sci 20, no. 3 (February 11, 2019). https://doi.org/10.3390/ijms20030751.Full Text Link to Item
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Keeler, Allison M., Marina Zieger, Sophia H. Todeasa, Angela L. McCall, Jennifer C. Gifford, Samantha Birsak, Sourav R. Choudhury, Barry J. Byrne, Miguel Sena-Esteves, and Mai K. ElMallah. “Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease..” Hum Gene Ther 30, no. 1 (January 2019): 57–68. https://doi.org/10.1089/hum.2018.016.Full Text Link to Item
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Borel, Florie, Huaming Sun, Marina Zieger, Andrew Cox, Brynn Cardozo, Weiying Li, Gabriella Oliveira, et al. “Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema..” Proc Natl Acad Sci U S A 115, no. 11 (March 13, 2018): 2788–93. https://doi.org/10.1073/pnas.1713689115.Full Text Link to Item
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McCall, Angela L., Jeffrey Salemi, Preeti Bhanap, Laura M. Strickland, and Mai K. Elmallah. “The impact of Pompe disease on smooth muscle: a review..” J Smooth Muscle Res 54, no. 0 (2018): 100–118. https://doi.org/10.1540/jsmr.54.100.Full Text Link to Item
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“Erratum for “Pediatric Obstructive Sleep Apnea in High-Risk Populations: Clinical Implications”.” Pediatric Annals 46, no. 11 (November 1, 2017): e436–e436. https://doi.org/10.3928/19382359-20171018-05.Full Text
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“Erratum for “Pediatric Obstructive Sleep Apnea and Asthma: Clinical Implications”.” Pediatric Annals 46, no. 11 (November 1, 2017): e436–e436. https://doi.org/10.3928/19382359-20171018-04.Full Text
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ElMallah, Mai, Evan Bailey, Michelle Trivedi, Ted Kremer, and Lawrence M. Rhein. “Pediatric Obstructive Sleep Apnea in High-Risk Populations: Clinical Implications..” Pediatr Ann 46, no. 9 (September 1, 2017): e336–39. https://doi.org/10.3928/19382359-20170815-01.Full Text Link to Item
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Trivedi, Michelle, Mai ElMallah, Evan Bailey, Ted Kremer, and Lawrence M. Rhein. “Pediatric Obstructive Sleep Apnea and Asthma: Clinical Implications..” Pediatr Ann 46, no. 9 (September 1, 2017): e332–35. https://doi.org/10.3928/19382359-20170815-03.Full Text Link to Item
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Keeler, A. M., M. K. ElMallah, and T. R. Flotte. “Gene Therapy 2017: Progress and Future Directions..” Clin Transl Sci 10, no. 4 (July 2017): 242–48. https://doi.org/10.1111/cts.12466.Full Text Link to Item
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Keeler, Allison M., Donghai Liu, Marina Zieger, Lang Xiong, Jeffrey Salemi, Karl Bellvé, Barry J. Byrne, David D. Fuller, Ronghua ZhuGe, and Mai K. ElMallah. “Airway smooth muscle dysfunction in Pompe (Gaa-/- ) mice..” Am J Physiol Lung Cell Mol Physiol 312, no. 6 (June 1, 2017): L873–81. https://doi.org/10.1152/ajplung.00568.2016.Full Text Link to Item
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Stoica, Lorelei, Allison M. Keeler, Lang Xiong, Michael Kalfopoulos, Kaitlyn Desrochers, Robert H. Brown, Miguel Sena-Esteves, Terence R. Flotte, and Mai K. ElMallah. “Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model..” Am J Respir Cell Mol Biol 56, no. 3 (March 2017): 405–8. https://doi.org/10.1165/rcmb.2016-0258LE.Full Text Link to Item
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Turner, S. M., M. K. ElMallah, A. K. Hoyt, J. J. Greer, and D. D. Fuller. “Ampakine CX717 potentiates intermittent hypoxia-induced hypoglossal long-term facilitation..” J Neurophysiol 116, no. 3 (September 1, 2016): 1232–38. https://doi.org/10.1152/jn.00210.2016.Full Text Link to Item
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Turner, Sara M. F., Aaron K. Hoyt, Mai K. ElMallah, Darin J. Falk, Barry J. Byrne, and David D. Fuller. “Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice..” Respir Physiol Neurobiol 227 (June 15, 2016): 48–55. https://doi.org/10.1016/j.resp.2016.02.007.Full Text Link to Item
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Loring, Heather S., Mai K. ElMallah, and Terence R. Flotte. “Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans..” Hum Gene Ther Methods 27, no. 2 (April 2016): 49–58. https://doi.org/10.1089/hgtb.2015.150.Full Text Link to Item
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Stoica, Lorelei, Sophia H. Todeasa, Gabriela Toro Cabrera, Johnny S. Salameh, Mai K. ElMallah, Christian Mueller, Robert H. Brown, and Miguel Sena-Esteves. “Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model..” Ann Neurol 79, no. 4 (April 2016): 687–700. https://doi.org/10.1002/ana.24618.Full Text Link to Item
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ElMallah, Mai K., David A. Stanley, Kun-Ze Lee, Sara M. F. Turner, Kristi A. Streeter, David M. Baekey, and David D. Fuller. “Power spectral analysis of hypoglossal nerve activity during intermittent hypoxia-induced long-term facilitation in mice..” J Neurophysiol 115, no. 3 (March 2016): 1372–80. https://doi.org/10.1152/jn.00479.2015.Full Text Link to Item
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Borel, Florie, Gwladys Gernoux, Brynn Cardozo, Jake P. Metterville, Gabriela C. Toro Cabrera, Lina Song, Qin Su, et al. “Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates..” Hum Gene Ther 27, no. 1 (January 2016): 19–31. https://doi.org/10.1089/hum.2015.122.Full Text Link to Item
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ElMallah, Mai K., Silvia Pagliardini, Sara M. Turner, Anthony J. Cerreta, Darin J. Falk, Barry J. Byrne, John J. Greer, and David D. Fuller. “Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines..” Am J Respir Cell Mol Biol 53, no. 3 (September 2015): 326–35. https://doi.org/10.1165/rcmb.2014-0374OC.Full Text Link to Item
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Bacewicz, Aleksandra, Wei Wang, Judy Ashouri, and Mai K. ElMallah. “Children with Chronic Lung Disease: Facilitating Smoking Cessation for their Caregivers..” J Community Health 40, no. 3 (June 2015): 409–13. https://doi.org/10.1007/s10900-014-9947-2.Full Text Link to Item
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Halawa, M., M. N. Abu-Hasan, and M. K. ElMallah. “Hereditary mucoepithelial dysplasia and severe respiratory distress.” Respiratory Medicine Case Reports 15 (April 8, 2015): 27–29. https://doi.org/10.1016/j.rmcr.2015.03.004.Full Text
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Falk, Darin J., Adrian Gary Todd, Sooyeon Lee, Meghan S. Soustek, Mai K. ElMallah, David D. Fuller, Lucia Notterpek, and Barry J. Byrne. “Peripheral nerve and neuromuscular junction pathology in Pompe disease..” Hum Mol Genet 24, no. 3 (February 1, 2015): 625–36. https://doi.org/10.1093/hmg/ddu476.Full Text Link to Item
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Abu-Hasan, Mutasim, Mai Elmallah, Dan Neal, and James Brookes. “Salivary Amylase Level in Bronchoalveolar Fluid as a Marker of Chronic Pulmonary Aspiration in Children..” Pediatr Allergy Immunol Pulmonol 27, no. 3 (September 1, 2014): 115–19. https://doi.org/10.1089/ped.2014.0348.Full Text Link to Item
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Elmallah, Mai K., Darin J. Falk, Sushrusha Nayak, Roland A. Federico, Milapjit S. Sandhu, Amy Poirier, Barry J. Byrne, and David D. Fuller. “Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice..” Mol Ther 22, no. 4 (April 2014): 702–12. https://doi.org/10.1038/mt.2013.282.Full Text Link to Item
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Fuller, David D., Mai K. ElMallah, Barbara K. Smith, Manuela Corti, Lee Ann Lawson, Darin J. Falk, and Barry J. Byrne. “The respiratory neuromuscular system in Pompe disease..” Respir Physiol Neurobiol 189, no. 2 (November 1, 2013): 241–49. https://doi.org/10.1016/j.resp.2013.06.007.Full Text Link to Item
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Falk, Darin J., Cathryn S. Mah, Meghan S. Soustek, Kun-Ze Lee, Mai K. Elmallah, Denise A. Cloutier, David D. Fuller, and Barry J. Byrne. “Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease..” Mol Ther 21, no. 9 (September 2013): 1661–67. https://doi.org/10.1038/mt.2013.96.Full Text Link to Item
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Elmallah, M. K., and L. Hendeles. “Delivery of medications by metered dose inhaler through a chamber/mask to young children with asthma.” Pediatric, Allergy, Immunology, and Pulmonology 25, no. 4 (December 1, 2012): 236–39. https://doi.org/10.1089/ped.2012.0197.Full Text
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ElMallah, Mai K., Darin J. Falk, Michael A. Lane, Thomas J. Conlon, Kun-Ze Lee, Nadeem I. Shafi, Paul J. Reier, Barry J. Byrne, and David D. Fuller. “Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9..” Hum Gene Ther Methods 23, no. 2 (April 2012): 148–56. https://doi.org/10.1089/hgtb.2012.009.Full Text Link to Item
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Elmallah, Mai K., Yasmeen Khan, Guenther Hochhaus, Jonathan J. Shuster, and Leslie Hendeles. “Systemic exposure to fluticasone MDI delivered through antistatic chambers..” J Allergy Clin Immunol 128, no. 5 (November 2011). https://doi.org/10.1016/j.jaci.2011.06.014.Full Text Link to Item
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ElMallah, Mai K., Sreekala Prabhakaran, and Sarah E. Chesrown. “Plastic bronchitis: resolution after heart transplantation..” Pediatr Pulmonol 46, no. 8 (August 2011): 824–25. https://doi.org/10.1002/ppul.21432.Full Text Link to Item
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Byrne, Barry J., Darin J. Falk, Christina A. Pacak, Sushrusha Nayak, Roland W. Herzog, Melissa E. Elder, Shelley W. Collins, et al. “Pompe disease gene therapy..” Hum Mol Genet 20, no. R1 (April 15, 2011): R61–68. https://doi.org/10.1093/hmg/ddr174.Full Text Link to Item
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Lee, Kun-Ze, Kai Qiu, Milapjit S. Sandhu, Mai K. Elmallah, Darin J. Falk, Michael A. Lane, Paul J. Reier, Barry J. Byrne, and David D. Fuller. “Hypoglossal neuropathology and respiratory activity in pompe mice..” Front Physiol 2 (2011). https://doi.org/10.3389/fphys.2011.00031.Full Text Link to Item
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El Mallah, M. K., A. J. Sands, F. A. Casey, B. G. Craig, and H. C. Mulholland. “Transcatheter occlusion of the patent ductus arteriosus: a comparison of two devices..” Ir J Med Sci 171, no. 3 (July 2002): 151–54. https://doi.org/10.1007/bf03170504.Full Text Link to Item
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El Mallah, M. K., U. Chakravarthy, and P. M. Hart. “Amblyopia: is visual loss permanent?.” Br J Ophthalmol 84, no. 9 (September 2000): 952–56. https://doi.org/10.1136/bjo.84.9.952.Full Text Link to Item
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Book Sections
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Kalfopoulos, Michael, Kaitlyn Wetmore, and Mai K. ElMallah. “Pathophysiology of Alpha-1 Antitrypsin Lung Disease.,” 1639:9–19, 2017. https://doi.org/10.1007/978-1-4939-7163-3_2.Full Text Link to Item
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Wang, W., A. Bacewicz, J. Ashouri, R. K. Elmallah, and M. K. ElMallah. “Secondhand smoke exposure in children with chronic lung disease.” In Nicotine Dependence, Smoking Cessation and Effects of Secondhand Smoke, 87–121, 2015.
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Conference Papers
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Zieger, Marina, Allison M. Keeler, Terence R. Flotte, and Mai K. ElMallah. “AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency..” In J Inherit Metab Dis, 42:870–77, 2019. https://doi.org/10.1002/jimd.12101.Full Text Link to Item
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Keeler, Allison M., Marina Zieger, Carson Semple, Ali Veinbachs, Guangping Gao, Robert H. Brown, Christian Mueller, and Mai K. ElMallah. “Respiratory Directed Gene Therapy to Silence SOD1 Prolongs Survival in the SOD1 ALS Mouse.” In Molecular Therapy, 27:422–422. CELL PRESS, 2019.Link to Item
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King, S., J. A. Sank, J. Hildebran, B. McLurkin, and M. K. ElMallah. “A RESPIRATORY THERAPIST-DRIVEN QUALITY IMPROVEMENT INITIATIVE IN THE EFFECTIVE MANAGEMENT OF AIRWAY CLEARANCE FOR THE PEDIATRIC PATIENT WITH CYSTIC FIBROSIS.” In Pediatric Pulmonology, 53:381–82. WILEY, 2018.Link to Item
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Lind, Lori A., Katherine A. Johnson, Angela L. McCall, Christian Mueller, Robert H. Brown, Teresa E. Lever, Mai K. Elmallah, and Nicole L. Nichols. “Impact of Intralingual AAVrh10-miRSOD1 Injection on Respiratory Function in SOD1(G93A) Mice.” In Faseb Journal, Vol. 32. FEDERATION AMER SOC EXP BIOL, 2018.Link to Item
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ElMallah, M. K., A. Keeler, M. Zieger, F. Borel, V. Semple, A. Veinbachs, R. Brown, and C. Mueller. “Respiratory Directed Gene Therapy Prolongs Survival In An Als Mouse Model.” In American Journal of Respiratory and Critical Care Medicine, Vol. 197. AMER THORACIC SOC, 2018.Link to Item
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Borel, Florie, Huaming Sun, Mai K. Elmallah, Andrew Cox, Brynn Cardozo, Weiying Li, Andrew M. Hoffman, Terence R. Flotte, and Christian Mueller. “Editing out Five SERPINA1 Paralogs to Create a New Mouse Model of Genetic Emphysema.” In Molecular Therapy, 25:117–117. CELL PRESS, 2017.Link to Item
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Keeler-Klunk, Allison M., Marina Zieger, Samantha Bircsak, Sourav Choudhury, Barry J. Byrne, Miguel Sena-Esteves, and Mai K. ElMallah. “Systemic Delivery of AAVB1-GAA Gene Therapy for Respiratory Pathology in Pompe Disease.” In Molecular Therapy, 25:28–29. CELL PRESS, 2017.Link to Item
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Keeler, A. M., M. Zieger, D. Liu, J. Salemi, R. ZhuGe, and M. K. ElMallah. “Respiratory And Airway Smooth Muscle Pathology In A Pompe Disease Mouse Model.” In American Journal of Respiratory and Critical Care Medicine, Vol. 195. AMER THORACIC SOC, 2017.Link to Item
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Lu, P., D. Liu, K. Bellve, K. Fogarty, M. K. ElMallah, L. Lifshitz, and R. ZhuGe. “Bitter Tastant 1,10-Phenanthroline Activates Tas2r-Gustducin Signaling To Relax Airways, Resulting In A Decrease In Airway Resistance In Vivo.” In American Journal of Respiratory and Critical Care Medicine, Vol. 195. AMER THORACIC SOC, 2017.Link to Item
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Borel, Florie, Brynn Cardozo, Andrew Cox, Weiying Li, Andrew Hoffman, Mai ElMallah, and Christian Mueller. “167. Genome Editing to Generate the First Mouse Model of Alpha-One Antitrypsin Deficiency, the Leading Cause of Genetic COPD.” In Molecular Therapy, 24:S65–S65. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)32976-8.Full Text
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Turner, Sara, Mai Elmallah, Aaron Hoyt, John Greer, and David Fuller. “Pretreatment with Ampakine CX717 Enhances Long-Term Facilitation (LTF) of Inspiratory Hypoglossal (XII) Bursting, But Only When Initial Burst Output is Low.” In Faseb Journal, Vol. 30. FEDERATION AMER SOC EXP BIOL, 2016.Link to Item
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Keeler, A. M., K. V. Desroshers, L. Xiong, and M. K. ElMallah. “Exercise Induced Respiratory Insufficiency In A Murine Model Of Very Long-Chain Acyl-Coa Dehydrogenase Deficiency.” In American Journal of Respiratory and Critical Care Medicine, Vol. 193. AMER THORACIC SOC, 2016.Link to Item
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Stoica, L., L. Xiong, M. Kalfopoulos, K. V. Desroshers, and M. K. ElMallah. “Characterization Of Pulmonary Mechanics Of The Sod1g93a Als Mouse Model.” In American Journal of Respiratory and Critical Care Medicine, Vol. 193. AMER THORACIC SOC, 2016.Link to Item
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ElMallah, Mai, David Stanley, and David Fuller. “Hypoglossal long term facilitation shifts the power spectral density to higher frequencies.” In Faseb Journal, Vol. 29. FEDERATION AMER SOC EXP BIOL, 2015.Link to Item
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Turner, Sara, Mai ElMallah, Darin Falk, Barry Byrne, and David Fuller. “Preferential Neuropathology in Respiratory-Related Motor Nuclei in Pompe Mice.” In Faseb Journal, Vol. 29. FEDERATION AMER SOC EXP BIOL, 2015.Link to Item
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Fuller, David, Mai Kamal Elmallah, Heather Ross, Anthony Cerreta, Manuela Corti, Barb Smith, Amy Poirier, Ronald Mandel, and Barry Byrne. “Selective knockout of acid alpha-glucosidase GAA gene expression in the medulla alters breathing.” In Faseb Journal, Vol. 28. FEDERATION AMER SOC EXP BIOL, 2014.Link to Item
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ElMallah, M. K., S. Pagliardini, S. M. Turner, J. J. Greer, B. J. Byrne, and D. D. Fuller. “Ampakines Enhance Respiratory Motor Output In A Murine Model Of Pompe Disease.” In American Journal of Respiratory and Critical Care Medicine, Vol. 189. AMER THORACIC SOC, 2014.Link to Item
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Falk, D. J., A. G. Todd, M. S. Soustek, D. C. Cloutier, M. ElMallah, D. D. Fuller, and B. J. Byrne. “P.17.9 AAV9 vector encoding hGAA improves synaptic pathology of the neuromuscular junction in Pompe disease.” In Neuromuscular Disorders, 23:829–829. Elsevier BV, 2013. https://doi.org/10.1016/j.nmd.2013.06.659.Full Text
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ElMallah, Mai Kamal, Milap S. Sandhu, Kun-Ze Lee, David M. Baekey, and David D. Fuller. “Apnea Alters the Pattern of Intermittent Hypoxia-Induced Long Term Facilitation in Anesthetized Mice.” In Faseb Journal, Vol. 27. FEDERATION AMER SOC EXP BIOL, 2013.Link to Item
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Sandhu, Milap Singh, Mai K. Elmallah, Michael A. Lane, Paul J. Reier, John J. Greer, and David D. Fuller. “Ampakines induce a persistent increase in phrenic motor output in rats with cervical spinal cord injury.” In Faseb Journal, Vol. 27. FEDERATION AMER SOC EXP BIOL, 2013.Link to Item
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Fuller, David, Mai Elmallah, Darin Falk, Lee Ann Lawson, A Daniel Martin, Barbara Smith, and Barry Byrne. “The importance of targeting the central nervous system for treatment of respiratory insufficiency in Pompe disease.” In Molecular Genetics and Metabolism, 108:S41–S41. Elsevier BV, 2013. https://doi.org/10.1016/j.ymgme.2012.11.089.Full Text
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Chakravarthy, U., M. El Mallah, and P. M. Hart. “Amblyopia: Is visual loss permanent..” In Investigative Ophthalmology & Visual Science, 41:S705–S705, 2000.Link to Item
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- Teaching & Mentoring
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Recent Courses
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